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Study of BMN 351 given by intravenous infusion to evaluate safety and tolerability in patients with Duchenne Muscular Dystrophy

Fast replyInvestigationalNo placebo
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What is this trial about?

A plain-language summary of the goals, design and what participants do

This study focuses on testing a new medication called BMN 351 in patients with Duchenne Muscular Dystrophy, a genetic condition that causes progressive muscle weakness and loss of muscle tissue. The purpose of this research is to evaluate how safe the medication is and how well patients can tolerate different dose levels when given through intravenous infusion.

The study will test BMN 351, which is a special type of molecule called a phosphorothioate oligonucleotide that targets a specific part of genetic material called exon 51. The medication is given as a concentrated solution that is diluted and administered directly into the bloodstream through an intravenous line. During the study, participants will receive multiple doses of the medication, and researchers will monitor how the body processes the drug.

Throughout the study, researchers will collect information about any side effects that may occur and perform various medical tests to ensure participant safety. They will also measure how the medication moves through the body and how it affects muscle tissue. This is an open-label study, which means all participants will receive the active medication, and the dose will be gradually increased over time to find the most appropriate amount.

The research process

The trial runs in 5 steps – from screening to follow-up. Each step says what happens and what the team monitors.

  1. Step 1

    Initial assessment and screening

    Confirmation of Duchenne muscular dystrophy diagnosis through genetic testing

    Basic health checks including urinalysis and physical examination

    Verification of ability to walk independently and complete a 10-meter walk/run test in 8 seconds or less

    Confirmation of stable corticosteroid or vamorolone treatment for at least 12 weeks prior

  2. Step 2

    Treatment initiation

    Start of BMN 351 administration through intravenous infusion

    The medication will be given as a concentrate that is diluted for infusion into a vein

    Different dose levels will be tested to determine safety and effectiveness

  3. Step 3

    Regular monitoring during treatment

    Regular physical examinations throughout the study period

    Safety laboratory tests to monitor health status

    Heart monitoring through ECG (electrical heart activity recording)

    Heart ultrasound (echocardiography) examinations

    Blood and urine samples to measure medication levels

    Muscle tissue analysis to check medication concentration

  4. Step 4

    Ongoing medication requirements

    Continuation of current corticosteroid or vamorolone treatment

    Dose adjustments of current medications only if needed due to weight changes

    The study will continue until approximately August 2026

  5. Step 5

    Safety monitoring

    Continuous monitoring for any side effects or health changes

    Regular assessment of breathing function to ensure no ventilation support is needed

    Documentation of any unexpected health events during the study period

Who can join the trial?

6 criteria

  • Must be a male child aged between 4 and 10 years old at the time of initial screening
  • Must have a confirmed diagnosis of Duchenne muscular dystrophy with a specific genetic mutation that can be treated with exon 51 skipping (this will be verified by a genetic counselor)
  • Must be able to walk independently without using any walking aids and complete a 10-meter walk/run test in 8 seconds or less
  • Must not need breathing support devices during the day and should not be expected to need them within the next year
  • Must be currently taking oral corticosteroids or vamorolone (medications that help manage muscle weakness) at a stable dose for at least 12 weeks before starting the study. The dose can only be adjusted for weight changes during the study
  • Must have normal urine test results during screening (a small amount of protein in urine is acceptable)

Who cannot join the trial?

13 criteria

  • History of any medical condition that would make the patient unsuitable for the study, as determined by the study doctor
  • Prior participation in any other clinical trial within 30 days before starting this study
  • Female patients (study is only open to male participants)
  • Age outside the children and adolescents range
  • Known allergies to the study medication or similar substances
  • Presence of other significant medical conditions that could interfere with the study results
  • Unable to follow study procedures or attend required study visits
  • Currently taking medications that could interact with the study drug
  • Previous treatment with gene therapy or cell therapy
  • Significant abnormal laboratory test results that could affect safety
  • Major surgery within 3 months before starting the study
  • Active infections or other serious illnesses
  • Mental conditions that could affect the ability to provide informed consent or follow study procedures
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Investigated drugs

BMN 351 is an investigational medication administered through intravenous (into the vein) injection. It is being studied as a potential treatment for Duchenne Muscular Dystrophy (DMD). This medication is designed to help address the underlying cause of DMD, a genetic condition that causes progressive muscle weakness. The study aims to understand how safe the medication is and how well patients tolerate it when given at different dose levels.

What is already known about the treatment

BMN 351 - An investigational drug administered through intravenous infusion, currently being studied in Phase 1/2 clinical trials for the treatment of Duchenne Muscular Dystrophy (DMD). The medication is being evaluated for its safety, tolerability, and effectiveness in patients with DMD, though its exact mechanism of action at the molecular level is still under investigation as part of the ongoing clinical research. As an experimental therapeutic agent, BMN 351 represents a new potential treatment option in the field of rare genetic disorders, specifically targeting the progressive muscle degeneration characteristic of DMD, with researchers currently studying different dose levels to determine optimal therapeutic effectiveness.

Investigated diseases

Duchenne Muscular Dystrophy - A genetic disorder that causes progressive muscle weakness and degeneration. The condition primarily affects boys and begins in early childhood, typically between ages 2 and 6. The disease is characterized by gradual weakening of muscles, starting in the legs and pelvis before affecting other parts of the body. Children with this condition often have difficulty with basic movements like walking, running, and jumping. The muscle weakness occurs because the body cannot produce a protein called dystrophin, which is essential for building and maintaining healthy muscle tissue. As the disease progresses, it affects various muscle groups throughout the body.
Trial detailsLast updated 2 Oct 2026
Age0-17PhasePhase I/IITrial ID2023-506737-30-00Protocol code351-201Estimated enrolment18 patientsSponsorBiomarin Pharmaceutical Inc.

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